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BW-40
- Development stage
- Unknown
- Lead developer
- Shanghai Argo Biopharmaceutical
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
BW-40 is a GalNAc-conjugated small interfering RNA (siRNA) therapeutic candidate developed by Shanghai Argo Biopharmaceutical (Argo Biopharma) for the treatment of rare diseases, specifically acute hepatic porphyria (AHP). Utilizing the company's proprietary RNAi platform, BW-40 is designed to target and silence the expression of the ALAS1 (5'-aminolevulinate synthase 1) gene in hepatocytes. By degrading ALAS1 mRNA, the drug reduces the production of the ALAS1 enzyme, which is the rate-limiting step in the heme biosynthetic pathway. This reduction prevents the pathological accumulation of neurotoxic heme intermediates, such as aminolevulinic acid (ALA) and porphobilinogen (PBG), which are responsible for the acute neurovisceral attacks and chronic symptoms associated with AHP. BW-40 is administered via subcutaneous injection and is currently in Phase 1 clinical development to evaluate its safety, tolerability, and pharmacodynamic effects in humans.
- Other names
- ALAS1-siRNAALAS-1-siRNAALAS 1-siRNA
02
Targets
ALAS1 (5-aminolevulinate synthase 1)
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